TMA live discussion

Drs. Jerry Mendell and Brian Kaspar are investigators in the follistatin gene therapy trial for inclusion-body myositis patients now underway at Nationwide Children’s Hospital. Several IBM patients have been treated, along with a number of Becker muscular dystrophy patients. Results in the Becker muscular dystrophy patients are encouraging, and show promise for sIBM patients. The Becker patients received a slightly higher dose of the treatment than the first sIBM patients. The results in the Becker patients might allow the next 6 sIBM patients to be treated with the highest dose of the treatment. This should produce better results in the sIBM patients as well. Approval is now being sought from federal regulatory bodies to allow the higher dose treatment. This historic treatment, pioneered in both mice and non-human primates with simulated sIBM, has prompted many questions from IBM patients.

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